ArticleJournal of kidney cancer and VHL2025
Long-term Safety and Efficacy of Belzutifan in Von Hippel-Lindau Syndrome: A VHL Coordinating Care Center Experience.
Article in Journal of kidney cancer and VHL, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
- Clinical outcomes of HIF-2α inhibition in Von Hippel-Lindau disease: a multicenter real-world study.The oncologist · 2026Article
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Authors and funding
16 authors.
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Abstract
Von Hippel-Lindau (VHL) disease is a rare inherited syndrome characterized by benign and malignant neoplasms. Belzutifan, a HIF-2α inhibitor, was approved for the treatment of VHL-associated neoplasms. As a first-in-class agent, understanding tolerability and efficacy outside of a clinical trial setting and optimizing the management of adverse events (AEs) is important. We conducted a retrospective analysis of VHL patients ≥18 years old, treated with belzutifan at Vanderbilt University Medical Center, between November 2018 and December 2024. Clinical data and AEs were collected. Primary endpoint was safety; secondary endpoints included dose reduction, treatment interruption, treatment discontinuation, time to anemia onset, time to dose reduction, tumor shrinkage, objective response (per RECIST 1.1), and need for subsequent VHL-related procedures. Among 25 patients, with a median follow-up of 35.0 months, any-grade AEs occurred in 23 (92%) patients; anemia was the most frequent (64%, no grade ≥ 3). The median time to anemia onset was 3.7 months. Treatment interruption happened in 80% of the patients. The dose reduction was needed in 15 (60%) patients, with a median time of 6.8 months, and the median final dose was 80 mg. Tumor shrinkage occurred in 89% of RCC patients, 80% of CNS hemangioblastoma, and 80% of pancreatic neuroendocrine tumors (pNET). Overall, four (20%) patients experienced the progression of the disease. During follow-up, three (12%) patients required new VHL-related procedures. These findings support the long-term safety and efficacy of belzutifan in VHL disease, underscore the utility of dose reduction for AE management while demonstrating durable disease control, and a low incidence of interventional procedures.
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